Hemophilia A
Conditions
Keywords
Cost, Clinical Outcomes, Quality of Life, On Demand Treatment, Secondary Prophylaxis
Brief summary
This study is a prospective, single center, observational, 2-cohort study of adult patients with severe Hemophilia A. There is no randomization procedure and all patients will be treated as per usual clinical practice. Patients will be followed up for 18 months after enrolment.
Detailed description
The primary therapy for hemophilia is coagulation factor replacement, given either episodically on demand (OD) for the treatment of acute bleeds or prophylactically to prevent them. The last years there has been an interest to study the comparative effectiveness of OD vs. SP (Secondary Prophylaxis) treatment strategies so as to create an evidence based platform to guide both clinical decision making and quantify the economic aspects of each treatment alternative. In Greece relevant information is lacking, thus it is imperative to study how patients on different treatment strategies are managed in a real-life setting in Greece, including the main factors associated with increased health care utilization, clinical success and patient well being.
Interventions
OTHERSurvey
Recombinant antihemophilic factor VIII (FVIII) on Secondary Prophylaxis treatment scheduled according to daily routine
Sponsors
Bayer
Study design
Observational model
COHORT
Time perspective
PROSPECTIVE
Eligibility
Sex/Gender
MALE
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Patients diagnosed with severe Hemophilia A. * Age ≥18 years old * Patients able to read and write * Patients receiving Factor VIII substitute therapy, either on-demand or as secondary prophylaxis. * Patients under the same therapeutic strategy (On Demand or Secondary Prophylaxis) for at least 6 months prior to enrolment.
Exclusion criteria
* Patients that have developed inhibitors against factor VIII. * Patients participating in an investigational program with interventions outside of routine clinical practice.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Annual direct medical cost of specialized hemophilia centre for patients with the on-demand strategy | At 18 months |
| Annual direct medical cost of specialized hemophilia centre for patients with secondary prophylaxis | At 18 months |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| Severity of joint bleed episodes for overall patients. | At 18 months | — |
| Severity of joint bleed episodes by cohort for patients. | At 18 months | — |
| Mean cost per joint bleed episodes for overall patients | At 18 months | — |
| Number of joint bleed episodes for overall patients | At 18 months | — |
| Mean EQ-VAS score as reported from patients at the time of a major bleed. | At 18 months | EQ-VAS score is Standardized questionnaire for hemophilia |
| Mean EQ-5D score as reported from patients at the time of a major bleed. | At 18 months | EQ-5D The EuroQol standardized instrument of health outcome |
| Mean change from baseline in Quality of Life scores derived from the Haem-A-Qol and EQ-5D questionnaires | Baseline and at 18 months | Haem-A-Qol Specific questionnaire for hemophilia and EQ-5D The EuroQol standardized instrument of health outcome |
| Mean cost per joint bleed episodes by cohort for patients | At 18 months | — |
| Number of joint bleed episodes by cohort for patients. | At 18 months | — |
Countries
Greece
Outcome results
None listed